A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
100 Clinical Results associated with Cholgene Therapeutics, Inc.
0 Patents (Medical) associated with Cholgene Therapeutics, Inc.
100 Deals associated with Cholgene Therapeutics, Inc.
100 Translational Medicine associated with Cholgene Therapeutics, Inc.